E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The intended indication is Pulmonary Arterial Hypertension |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064911 |
E.1.2 | Term | Pulmonary arterial hypertension |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The main objective of the AC-065A303/GRIPHON OL study will be to assess the longterm safety and tolerability of ACT-293987 in patients with PAH. |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed informed consent form (ICF) prior to initiation of any study-mandated procedure. 2. Patients who have completed the double-blind studies AC-065A301 or AC-065A302 as scheduled per protocol, or, who have experienced a clinical worsening of PAH (adjudicated by the CEC) after their Week 16 assessments in AC-065A301 or AC-065A302 studies, and for whom a written approval to be transferred into this study has been obtained from the sponsor. 3. Women of child-bearing potential included in study AC-065A303 must use a reliable method of contraception (with a failure rate of less than 1% per year) until one month after end of study treatment. |
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E.4 | Principal exclusion criteria |
1. Any major violation of protocols AC-065A301 or AC-065A302. 2. Patients who have experienced a clinical worsening of PAH before their Week 16 assessment in study AC-065A301 or AC-065A302. 3. Females who plan to become pregnant during the study, or are breastfeeding. 4. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results, such as drug or alcohol dependence, or psychiatric disease. 5. Known hypersensitivity to ACT-293987 or any of the excipients. |
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E.5 End points |
E.5.1 | Primary end point(s) |
No primary efficacy endpoint is considered for this OL study. The primary endpoint for this study is safety and tolerability. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 63 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Study treatment for each patient lasts from his/her Visit 1 date until the end of the trial i.e., until whichever of the following occurs first: (i) the approval of ACT-293987 in this indication is obtained in the patient’s country, (ii) the sponsor decides to stop study AC-065A303/GRIPHON OL, or (iii) the patient, the investigator or the sponsor decides to discontinue study drug. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |